Tofa, would be nice if as cheap as Tofu

By Dr Irwin Lim, Rheumatologist

My twitter feed was abuzz with the Presidential election results (congrats, Mr Obama).

The other item making the rounds and retweets was the news that the Pfizer drug, Tofacitinib, had been approved by the US Food & Drug Administration as a treatment for moderate to severe rheumatoid arthritis in patients who can’t take Methotrexate or who haven’t been helped by it.

This is exciting but expected news. There's been a buzz about Tofacitinib for a couple of years and I thought you might be interested in a rheumatologist's perspective.

Please note that I was not involved in any of the clinical trials and therefore, do not have personal experience with the use of Tofacitinib.

Why Tofacitinib is a potential blockbuster drug:

• It's a new ORAL agent, in the era of biologic medications (usually given as subcutaneous injections or intravenous infusions).
• A novel action. Tofacitinib belongs to a new class of agents called JAK inhibitors. These drugs block enzymes involved in the signalling systems of cells, including the signalling systems involved with many cytokines.
• It should be a relatively CHEAP medication to manufacture unlike the other biologic medications such as the TNF inhibitor.
• There is good quality scientific trial evidence of it's effectiveness in a variety of scenarios: as mono therapy, in patients who haven't responded well enough to Methotrexate, and in those who haven't responded well enough to TNF inhibitor therapy.

But I, like many of my colleagues, have many questions.

The field of new therapies for Rheumatoid Arthritis is crowded (which is a good thing) but it's also confusing and we aren't quite sophisticated enough to be able to personalise the choice of medication to each individual patient.

For those who don't respond adequately to traditional DMARDs, the biologic agents are used (in countries that can afford them). In Australia, we have access to 5 different TNF inhibitor medications as well as 3 other drugs with different mechanisms of actions: Abatacept (Orencia), Rituximab (Mabthera) & Tocilizumab (Actemra). We will soon have biologic drug no.9.

Which patient should receive which drug?

They all seem to be effective in the majority of patients. But there are still significant numbers of patients who don't respond well enough. They all have potentially worrying side effects but overall have been tolerated better than expected, as can be seem by the many drug registries set up to track side effects.

Where do I use Tofacitinib?

It's a new drug and has to battle familiarity. After a decade, most rheumatologists are quite comfortable using the older biological drugs.

Does price matter?

The 8 biologic drugs I currently have access to are all EXPENSIVE. In Australia, I think costs are around $25000 a year per patient. Yes, each year.

Now, Tofacitinib is meant to be a much simpler, and therefore cheaper drug to manufacture than the other biologic drugs mentioned.

This doesn't however mean that it will be cheap. It's only right that pharmaceutical companies recuperate their development costs and make some profit. Otherwise, new medication development will be stymied.

So, one big question is how will Tofacitinib be PRICED?

Given all countries are struggling to contain medical costs, and the use of these expensive medications are regulated, the price could very well determine how Tofacitinib is used, and indeed, how much is used.